hemophilia
Pfizer Discontinues Development, Commercialization of Hemophilia B Gene Therapy Beqvez
Pfizer opted to discontinue the product for "several reasons," including limited interest from patients and physicians in gene therapies for hemophilia.
Be Biopharma Raises $92M in Series C Financing to Advance Engineered B-Cell Therapies
The firm will use the proceeds to advance BE-101 for hemophilia B through clinical proof-of-concept testing and BE-102 for hypophosphatasia into the clinic.
Sangamo Regains Rights to Hemophilia A Gene Therapy After Pfizer Ends Partnership
Sangamo is seeking new partners to continue advancing giroctocogene fitelparvovec, which reported positive Phase III results this year.
Pfizer's Beqvez Bests Standard Hemophilia B Care in Trial, but Unclear Why Not All Sustain Benefits
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Patients had fewer bleeds after treatment with Beqvez compared to standard treatment, but questions remain about which patients have enduring benefits.
During a panel discussion at the National Society of Genetic Counselors meeting, genetic counselors discussed the risks and benefits of gene therapies.